Hydrocortison (1) – Alkindi®

• Clinical trials
  • The Infacort 003 study included 24 children under the age of 6 with adrenal insufficiency who were already receiving hormone replacement therapy with hydrocortisone (not Alkindi®) prior to enrolment in the study.
  • In the Infacort 004 study, treatment with hydrocortisone (Alkindi®) was continued as long-term therapy for 18 of the 24 children.

Newborns, children and adolescents (from birth to under 18 years of age) with adrenal insufficiency

• An additional benefit is not proven.
• Overall, for this reason, the G-BA concludes that additional benefit from hydrocortisone (Alkindi®) is not proven for newborns, children and adolescents (from birth to under 18 years of age) with adrenal insufficiency.
• To demonstrate the additional benefit of hydrocortisone (Alkindi®), the pharmaceutical manufacturer has submitted the two single-arm, single-centre studies Infacort 003 and Infacort 004.
• As the clinical development programme for Alkindi® did not include any comparative studies, such as an investigation of Alkindi® versus hydrocortisone in adult patients with adrenal insufficiency, evidence transfer for paediatric medicinal products within the meaning of Article 2(4) of Regulation (EC) No. 1901/2006 of the European Parliament and of the Council on paediatric medicinal products, in accordance with Section 5(5a) of the AM-NutzenV, is not possible in the present case for deriving the additional benefit of hydrocortisone (Alkindi®).
• Conclusion
  • To demonstrate the additional benefit of hydrocortisone (Alkindi®), the pharmaceutical manufacturer has submitted the two single-arm, single-centre studies, Infacort 003 and Infacort 004.
  • These studies formed the basis for the granting of the Paediatric Use Marketing Authorisation (PUMA) by the European regulatory authority.
  • However, the two non-comparative studies, Infacort 003 and Infacort 004, do not allow for a weighing up of the benefits and harms of hydrocortisone (Alkindi®) against the appropriate comparator therapy and cannot therefore be used to establish any additional benefit of hydrocortisone (Alkindi®) under Section 35a of the German Social Code, Book V (SGB V).
  • As the clinical development programme for Alkindi® did not include any comparative studies, such as an investigation of Alkindi® against hydrocortisone in adult patients with adrenal insufficiency, evidence transfer for paediatric medicinal products within the meaning of Article 2(4) of Regulation (EC) No. 1901/2006 of the European Parliament and of the Council on medicinal products for paediatric use, in accordance with Section 5(5a) of the AM-NutzenV, is not possible in the present case for the derivation of the additional benefit of hydrocortisone (Alkindi®).

Courtesy translation only, please refer to the German original.